SUPPLY CHAIN ARTICLES
-
mRNA Vaccines: Key Considerations For Development & Manufacturing
As we wind down National Immunization Awareness Month, this article looks at the complexities around developing and manufacturing mRNA vaccines and how you should address those complexities. Topics include delivery, process validation, long-term effectiveness, GMP, regulatory approval, and more.
-
Therapeutic Oligonucleotides: Regulations & Quality Standards
Oligonucleotides have surfaced in the past few years as promising therapeutic agents to treat diseases such as neurodegenerative disorders, respiratory disorders, cancer, and diabetic retinopathy. This article recaps a recent USP workshop where participants from regulatory agencies, industry, and academia gathered to discuss best practices and perspectives.
-
Inside Alnylam's Playbook For High Volume siRNA Production
As one of the first companies to establish a chemoenzymatic ligation platform, Alnylam's Maines and Nechev are in the perfect position to espouse wisdom on the practical aspects and remaining challenges of implementing enzymatic ligation at a large scale. Here in this installment, Maines and Nechev outline what they’ve learned thus far about the science of enzymatic ligation, while also paying credence to the unknowns and existing barriers that, as a frontrunner, Alnylam will inevitably (but willingly) be tasked with facing in the future.
-
Bridging The Atlantic: A European Executive's Guide To Building A Successful U.S. RNA Business (Pt. 2)
European RNA biotech leaders can accelerate U.S. success by aligning fundraising, partnerships, manufacturing, hiring, and regulatory strategy from day one.
-
Multidisciplinary mRNA: What Can We Learn From Other CGTs?
Overall, there are four high-level best practices/mindsets I proposed during a recent presentation that I believe will be crucial for us to bring some talented RNA therapeutic role models to the forefront. But there is one best practice that I think is worth emphasizing more than the others.
-
Picasso, Pick-Up Lines, & Personalized Medicine: mRNA's Path Forward
Overall, my presentation on what the mRNA industry can learn from the artist Picasso singled out a few specific areas from which I felt our industry could garner greater inspiration and operational prowess. But given how “collaborative” many of these sources of inspiration are, I’ve introduced each of my takeaways with a surprisingly relevant (albeit bad) art-themed pick-up line. After all, if there’s one sector that knows it takes more than one to “tango,” it’s the mRNA industry.
ARTICLES, APP NOTES, CASE STUDIES, & WHITE PAPERS
-
Explore innovative solutions for CDMO's striving to remain competitive as the biopharmaceutical market grows and evolves.
-
Read through the results of our efforts to produce an optimal protocol for the transient transfection of hMSCs with pDNA or mRNA using non-viral means.
-
Prioritize operational efficiency over early-stage flexibility to scale advanced therapies. Standardized workflows and qualified shipping lanes reduce variability for predictable late-phase success.
-
We report on the use of a novel lipid nanoparticle (LNP) reagent in a validated protocol to achieve successful complex gene editing in primary T cells with high efficiency while maintaining high cell viability.
-
Explore ongoing work to harness different types of HDR templates in order to build cutting-edge systems with the potential to enable point-of-care cell and gene therapy manufacture.
-
CRISPR's potential depends on more than biology. Learn how advances in delivery, safety, manufacturing, and regulation are enabling clinical translation at scale.
-
The single guide RNA (sgRNA) is one of the key components of successful CRISPR gene editing. Here we cover all the important aspects of sgRNA and Synthego’s sgRNA products.